Angelman syndrome (AS) is a rare neurodevelopmental disorder caused by the loss of function of the maternally inherited ubiquitin E3 ligase UBE3A gene. AS is characterized by severe symptoms, including lack of speech, epilepsy, developmental and motor skills delays, sleep disturbances and cognitive impairment. Currently, no treatments are available for this disease.
Pilatus Biosciences Inc. has announced a clinical trial collaboration with F. Hoffmann-La Roche Ltd. in support of Pilatus’ upcoming first-in-human phase I trial evaluating PLT-012 in combination with atezolizumab in patients with hepatocellular carcinoma (HCC).
F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have divulged monoglyceride lipase (MGLL; MAGL) inhibitors reported to be useful for the treatment of cancer, amyotrophic lateral sclerosis, multiple sclerosis, pain, neuroinflammation, neurodegeneration, Alzheimer’s disease and inflammatory bowel disease, among other disorders.
F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have discovered new heteroarylphenyl ether derivatives acting as cyclin-dependent kinase CDK8/cyclin C inhibitors. As such, they are reported to be useful for the treatment of graft-vs.-host disease, transplant rejection, multiple sclerosis, rheumatoid arthritis, atopic dermatitis, psoriasis, amyotrophic lateral sclerosis and stroke, among others.
Alterations in the GABAergic pathway in the brain contribute to the pathophysiology of neurodevelopmental disorders, including autism spectrum disorder and Angelman syndrome. Targeting the GABA-A receptor α5 subunit (GABRA5) with positive allosteric modulators represents a new therapeutic strategy for the treatment of these conditions.
F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have divulged bifunctional epoxide hydrolase 2 (EPHX2; sEH) inhibitors reported to be useful for the treatment of pain, diabetic retinopathy and neurodegeneration. An exemplified compound (Ex 23 pg 78, claim 18) inhibited human sEH activity (IC50=0.5 nM) using Rapidfire mass spectrometry (RFMS) assays (WO 2025125135).
F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have identified new macrocyclic compounds acting as GTPase KRAS (G12C mutant) inhibitors reported to be useful for the treatment of cancer.
F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have patented 5-[2,7-diazaspiro[3.5]nonan-7-yl]-5-[4-phenoxyphenyl]hexahydropyrimidine-2,4,6-trione derivatives acting as matrix metalloproteinase-9, (MMP-9) inhibitors potentially useful for the treatment of dry eye.
Scientists at F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have identified macrocyclic compounds acting as GTPase KRAS (G12D mutant) and/or (G13D mutant) inhibitors reported to be useful for the treatment of cancer.
Scientists at ETH Zürich, F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have synthesized fluorescent probes acting as monoglyceride lipase (MGLL; MAGL) inhibitors reported to be useful for diagnostic imaging of MAGL.
